Movement disorders and mRNA therapy in Arginosuccinic aciduria
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Dr Sonam Gurung and Dr Julien Baruteau discuss movement disorders in Arginosuccinic aciduria and explain how recent work with mRNA therapy shows potential as a treatment in this condition.
The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduria
Gurung et al
https://doi.org/10.1002/jimd.12691
mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria
Gurung et al
https://doi.org/10.1126/scitranslmed.adh1334
Movement disorders and mRNA therapy in Arginosuccinic aciduria